BPC December 03 update

Biotech week in review; Clinical data readouts to watch in December

Weekly watchlist

Just four weeks remain in the trading year, yet a number of key clinical readouts are still slated for release before the end of 2020. We list a selection below. Readers may also view our preview of PDUFA and other regulatory events, including FDA Advisory Committee meetings for two COVID-19 vaccine candidates, scheduled for December.

First, let’s review the week that was with price-moving events.

COVID-19 VACCINE:

Moderna, Inc. (NASDAQ:MRNA) shares closed the week up 20% to $152.52. The company announced further data from its COVID-19 vaccine Phase 3 trial of mRNA-1273 conducted on 196 cases, confirming high efficacy observed at the first interim analysis, indicating a vaccine efficacy of 94.1%. The data showed that all 30 severe cases of COVID-19 in the trial occurred in the placebo group with none occurring in the mRNA-1273 vaccinated group. The company also announced the submission to the FDA of a Emergency Use Authorization (EUA, which will be reviewed by the FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday, December 17.

Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) shares closed the week up 47% to $112.01. The company is in earlier Phase 1/2 development for its COVID-19 vaccine ARCT-021, which similar to Moderna and Biontech (BNTX) uses messenger RNA (mRNA) technology.

REGULATORY:

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced Thursday that the FDA approved oral, once-daily Orladeyo (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Shares closed Friday up 19% to $6.10.

Minerva Neurosciences, Inc. (NASDAQ:NERV) shares closed Wednesday down 26% to $2.89 following the release of feedback from the FDA regarding its drug roluperidone, in development for patients with schizophrenia. Its Phase 3 trial failed to meet the primary endpoint earlier this year, and in its update, it noted:

“The FDA cautioned that an NDA submission based on the current data from the Phase 2b and Phase 3 studies would be highly unlikely to be filed and that, at a minimum, there would be substantial review issues due to the lack of two adequate and well-controlled trials to support efficacy claims for this indication.”

CLINICAL DATA:

Sutro Biopharma, Inc. (NASDAQ: STRO) shares closed Friday up 33% to $22.65 following the release of data from its Phase 1 trial of STRO-002 in patients with ovarian cancer. Of the 31 evaluable patients, 1 patient achieved a complete response (CR) and 9 patients achieved a partial response (PR) - 3 confirmed and 6 unconfirmed. 23 patients (74%) achieved disease control at 12 weeks, while 18 patients (58%) achieved disease control at 16 weeks.

Ovid Therapeutics Inc. (NASDAQ:OVID) shares fell Wednesday 56% to $2.94 following news its Phase 3 Neptune trial of OV101 (gaboxadol) for the treatment of Angelman syndrome did not meet the primary endpoint.

---

Biotech clinical data readouts to watch in December:

Drug Stage Catalyst Market Cap

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan
Paroxysmal Nocturnal Hemoglobinuria (PNH)

PDUFA priority review PDUFA date under priority review May 14, 2021.
$3.6 billion

APRE – Aprea Therapeutics Inc.
APR-246 (eprenetapopt) and azacitidine
TP53 mutant Myelodysplastic syndromes (MDS)

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 28, 2020.
$124.4 million

ARPO – Aerpio Pharmaceuticals Inc.
Razuprotafib (AKB-9778)
Open-angle glaucoma (OAG)

Phase 2 Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.
$61.7 million

BLRX – BioLineRx Ltd.
BL-8040 in combination with KEYTRUDA - COMBAT trial
Pancreatic cancer

Phase 2 Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.
$60 million

GSK – GlaxoSmithKline PLC
Adjuvanted COVID-19 vaccine
COVID-19 vaccine

Phase 2b Phase 1/2 study interim results showed an immune response comparable to patients who recovered from COVID-19 in adults aged 18 to 49 years, but a low immune response in older adults. Phase 2b trial to commence February 2021. Phase 3 trial to commence 2Q 2021.
$95 billion

LYRA – Lyra Therapeutics Inc.
LYR-210 - LANTERN
Chronic rhinosinusitis / Chronic Sinusitis

Phase 2 Phase 2 trial did not meet primary endpoint - December 7, 2020.
$148.3 million

QURE – uniQure N.V.
AMT-061 (HOPE-B)
Hemophilia B

Phase 3 Phase 3 clinical hold announced December 21, 2020.
$1.7 billion

RCKT – Rocket Pharmaceuticals Inc.
RP-A501
Danon disease

Phase 1 Phase 1 promising early efficacy data released December 8, 2020 - low dose generally well-tolerated with manageable safety. One of two patients receiving high dose experienced drug product related severe adverse event.
$3.2 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 top-line data met primary endpoint - December 22, 2020. All primary endpoint responders were patients with BBS. Alstrom syndrome patients did not meet primary endpoint.
$1.6 billion

SNGX – Soligenix Inc.
SGX942 (dusquetide)
Oral Mucositis in Patients with Head & Neck Cancer

Phase 3 Phase 3 trial did not meet primary endpoint - December 22, 2020.
$50.4 million

SRPT – Sarepta Therapeutics Inc.
SRP-5051 (MOMENTUM)
Duchenne muscular dystrophy

Phase 1/2 Phase 2 interim data released December 7, 2020. At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings.
$7.6 billion

TGTX – TG Therapeutics Inc.
TG-1101 - ULTIMATE I
Multiple Sclerosis

Phase 3 Phase 3 trial met primary endpoint - December 10. 2020.
$6.5 billion

TNXP – Tonix Pharmaceuticals Holding Corp.
TNX-102 SL (Tonmya)
Fibromyalgia

Phase 3 Phase 3 top-line met primary endpoint - December 7, 2020. Data from second Phase 3 trial due 2H 2021.
$259.5 million

VTVT – vTv Therapeutics Inc.
Azeliragon
Mild Alzheimer’s disease with type 2 diabetes

Phase 2 Phase 2 top-line data did not meet primary endpoint - December 15, 2020.
$165.4 million